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The New England Journal of Medicine|November 18, 2024
CRISPR-Cas9 Gene Editing with Nexiguran Ziclumeran for ATTR CardiomyopathyMarianna Fontana, Scott D Solomon, Jessica Kachadourian, et al.
Amyloid : the International Journal of Experimental and Clinical Investigation : the Official Journal of the International Society of Amyloidosis|March 6, 2020
Quality of life outcomes in APOLLO, the phase 3 trial of the RNAi therapeutic patisiran in patients with hereditary transthyretin-mediated amyloidosisLaura Obici, John L Berk, Alejandra González-Duarte, et al.
Nano Letters|September 14, 2020
Cancer Immunotherapy via Targeting Cancer Stem Cells Using Vaccine NanodiscsAlireza Hassani Najafabadi, Jing Zhang, Marisa E Aikins, et al.
European Journal of Neurology|April 22, 2025
TTR Gene Screening Since the Advent of Biotherapies in France: A Nationwide Retrospective Survey Between 2018 and 2023Abd El Kader Ait Tayeb, Pauline Chazelas, Vianney Poinsignon, et al.
Human Mutation|August 8, 2015
PhenomeCentral: a portal for phenotypic and genotypic matchmaking of patients with rare genetic diseasesOrion J Buske, Marta Girdea, Sergiu Dumitriu, et al.
Orphanet Journal of Rare Diseases|October 17, 2021
A double-blind, placebo-controlled, randomized trial of PXT3003 for the treatment of Charcot-Marie-Tooth type 1AShahram Attarian, Peter Young, Thomas H Brannagan, et al.
Circulation|December 28, 2018
Effects of Patisiran, an RNA Interference Therapeutic, on Cardiac Parameters in Patients With Hereditary Transthyretin-Mediated AmyloidosisScott D Solomon, David Adams, Arnt Kristen, et al.
The New England Journal of Medicine|September 26, 2025
Nexiguran Ziclumeran Gene Editing in Hereditary ATTR with PolyneuropathyJulian D Gillmore, Ed Gane, Jörg Täubel, et al.
The British Journal of Dermatology|March 4, 2026
Targeting non-canonical NF-κB signalling in CYLD cutaneous syndrome by selective inhibition of IκB kinase alphaKirsty Hodgson, Joseph Inns, Gary Reynolds, et al.
Journal of Controlled Release : Official Journal of the Controlled Release Society|July 19, 2021
Vaccine nanodiscs plus polyICLC elicit robust CD8+ T cell responses in mice and non-human primatesAlireza Hassani Najafabadi, Zeynab Izadi Najaf Abadi, Marisa E Aikins, et al.
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